Lindsey Shapiro, PhD,  science writer—

A lab-made version of a plant enzyme prevented toxic huntingtin protein clumps — that drive Huntington’s disease from forming — in human cell and worm models of the neurodegenerative condition, a study shows. Called stromal processing peptidase (SPP), the enzyme is naturally present in plants’ chloroplasts, where sunlight is…

The benefits of Ingrezza (valbenazine) for people with Huntington’s disease — reductions in uncontrolled movements and gains in clinician- and patient-reported health — were seen two weeks after starting treatment and maintained for up to three months, according to new KINECT-HD trial analyses. Ingrezza was cleared last month…

Caregivers of people with Huntington’s disease want support programs that will help them in balancing their own care needs over time with those of the patient, often a family member. This theme emerged from caregiver focus groups conducted in the Netherlands, where support was considered effective if it would…

With early data on AMT-130 for Huntington’s disease reportedly looking promising, uniQure said it’s planning to meet early next year with regulatory authorities to discuss the late-stage development of its gene therapy candidate. By that time, more data on the ongoing Phase 1/2 clinical trials testing the therapy…

PTC Therapeutics’ Huntington’s disease treatment candidate PTC518 is well-tolerated and lowers blood levels of the huntingtin protein (HTT) in people with the neurodegenerative disorder. That’s according to interim three-month data from the Phase 2a PIVOT-HD trial (NCT05358717), which found that use of the investigational oral therapy did not…

Note: This story was updated May 16, 2023, to correct the duration of ANX005 infusions. Only the first loading dose of the therapy is given for 21 hours, with subsequent doses having a duration of four to five hours. The experimental therapy ANX005 safely leads to stabilization or improvements in…

The last patient has completed the final visit in the Phase 3 PROOF-HD trial testing oral pridopidine for Huntington’s disease. That means that the study’s main placebo-controlled part is done, according to Prilenia Therapeutics, pridopidine’s developer, which sponsored the nearly 18-month-long trial. Now, the first data are…

Enrollment is underway in a Phase 2 clinical trial testing Roche’s therapeutic candidate tominersen in people with early manifest Huntington’s disease and those in the prodromal phase of the disease, when hallmark disease symptoms are not yet present. The study, dubbed GENERATION HD2 (NCT05686551), aims…

Long-term treatment with Austedo (deutetrabenazine) safely led to sustained reductions in chorea for people with Huntington’s disease, including those who had switched from treatment with Xenazine (tetrabenazine). That’s according to about three years of follow-up data in the Phase 3 ARC-HD trial (NCT01897896), a study jointly conducted…

A data safety monitoring committee has recommended that enrollment of the highest dosing group resume in the European Phase 1/2 trial of AMT-130 in people with Huntington’s disease, but with additional safety measures in place. UniQure, the investigational treatment’s developer, announced in August that enrollment in…